Approval of Zolgensma good news for Fairview toddler
South Peace News
Health Canada’s approval of an expensive new gene-therapy treatment spells hope for a Peace Country toddler.
Max Sych, the son of Fairview town Councillor Bryarly Parker and Bowden Psych, was diagnosed with spinal muscular atrophy [SMA] in late November. SMA affects muscle development, including the ability to walk, swallow and even breathe. Max was diagnosed with Type 2 SMA.
Born at barely 25 weeks and weighing only 1 lb 11 oz, Max was extremely premature and spent the first months of his life receiving numerous blood transfusions, surgery, respiratory therapy and oxygen among other special care.
However, despite his difficult early life, he was developing normally for his adjusted age until about a year old when his parents noticed he was collapsing and starting to have trouble walking. Since his Nov. 24 diagnosis of SMA, which his family says actually has nothing to do with his prematurity, Max’s family and friends have been in a race against time to raise the $2.8 million they need to get him Zolgensma.
Zolgensma treats the rare genetic disorder by replacing the faulty gene that causes SMA in about one in every 6,000 babies. The treatment was first approved by the U.S. Food and Drug Administration in May 2019, followed by Japan and the European Union. It was approved by Health Canada Dec. 16 after undergoing a priority review.
In a Facebook announcement, Parker calls the approval of the drug “a massive win for the SMA community.”
She says she is very grateful for all of the families who came before hers and have fought for the treatment.
Funding for the drug has yet to be approved, although that could happen sometime in 2021. The provinces are currently negotiating a price for the treatment.
“Our understanding for what this means is Max just can’t go up to the hospital and get dosed,” Parker says.
“We will be continuing to fundraise for this because the sooner Max receives this treatment the better. This is a disease that gets worse and worse every day.”
For about the past two years, the main treatment for children in Alberta with SMA has been Spinraza, a drug which costs $375,000 a year for life and is given by spinal injection every few months. While Max has been approved for that drug under Alberta healthcare, it may not be as effective over time. Zolgensma is only a one-time injection.
The family has been working under the assumption Max needs to get the new treatment by his second birthday on Jan. 19 due to age restrictions. In other countries the medication is only authorized for children who weigh less than 21 kg and are under the age of two.
According to his aunt Maya Shirlene, while Health Canada has now lifted the age restriction, the weight restriction has not been lifted. Parker says it’s unclear at this time what that means for Max.
At least three other children in Alberta have been diagnosed with SMA. Reign Johnston, who also still needs the treatment, turns two in March. Two other children have already gotten the treatment; Harper Hanki got it thanks to a pharmaceutical company lottery and Kaysen Martin got it through fundraising.
At press time the GoFundMe set up to help Max get the treatment has raised $538,372 of the $2.8 million needed.
To follow Max’s journey or participate in an online auction, find Moving Mountains for Mighty Max on Facebook, @movingmountainsformightymax on Instagram, or visit the website https://www.mightymaxsych.org